We are engaged in research which is aimed at developing biotherapeutic molecules for use in the treatment of a class of genetic diseases known as lysosomal storage diseases. We have received approval from the National Institute of Health's Recombinant DNA Advisory Committee to initiate clinical trials to treat Gaucher disease by gene therapy. This protocol will involve delivery of an enzyme (which is deficient in this disease) by retroviral transduction of bone marrow cells. Other biomolecular strategies employing recombinant DNA technology are being studied for development of treatment modalities for human genetic disease.